{"id":"W4402969161","doi":"10.1016/j.omtn.2024.102349","title":"Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina","year":2024,"lang":"en","type":"article","venue":"Molecular Therapy — Nucleic Acids","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":11,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Domaine d’Intérêt Majeur Logiciels et Systèmes Complexes; Sorbonne Université; Fondation pour la Recherche Médicale; Institut National de la Santé et de la Recherche Médicale; AFM-Téléthon; Association Française contre les Myopathies; Agence Nationale de la Recherche; Fondation Voir et Entendre; Foundation Fighting Blindness","keywords":"Cas9; CRISPR; Genome editing; Computational biology; Guide RNA; Computer science; RNA; Base (topology); Retina; Genome; Biology; Genetics; Gene; Neuroscience","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003731458,0.0001893057,0.0001714775,0.00006674838,0.00004853479,0.00004995965,0.0002227903,0.0001088572,0.00003811421],"category_scores_gemma":[0.0000460046,0.0001360994,0.00008375312,0.0001619711,0.00008243899,0.000004927776,0.00007503285,0.0001135804,0.000002454008],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001261178,"about_ca_system_score_gemma":0.00004601115,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005706422,"about_ca_topic_score_gemma":0.0000164469,"domain_scores_codex":[0.9989111,0.00009250219,0.0002522583,0.0003307006,0.0001705888,0.0002428261],"domain_scores_gemma":[0.999528,0.0000231161,0.00003604153,0.0003276897,0.0000405846,0.00004455699],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.00005801326,0.00002599604,0.00003820885,0.00007695538,0.00005321881,0.00007641633,0.0002627447,0.0001444958,0.9938635,0.00002491661,0.001224807,0.004150758],"study_design_scores_gemma":[0.0004937169,0.0004031897,0.0006382862,0.00009374092,0.00001756667,0.00004110375,0.0003624458,0.0007078258,0.7898824,0.00002763595,0.2070817,0.0002504065],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9857402,0.008918841,0.003568019,0.0002639884,0.0002488432,0.0003872067,0.00003732443,0.0000236297,0.0008119551],"genre_scores_gemma":[0.9964682,0.0006056208,0.001499452,0.0002225327,0.0009141524,0.00004451619,0.00005076206,0.0000436476,0.0001511374],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.2058569,"threshold_uncertainty_score":0.5549973,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01028850280644513,"score_gpt":0.2708784636405886,"score_spread":0.2605899608341435,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}